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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IICognition Disorders, Cognitive Dysfunction, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, cognitive disorder, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorderActive, not recruitingPhase 2 / Phase 32026-09-21NCT05208281
The Happiest Baby, Inc. SNOO Postmarket Surveillance StudyDeath, Infant Death, Pathologic Processes, Pathological Conditions, Signs and SymptomsRecruitingNot stated2026-09-15NCT06361303
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 2 / Phase 32026-09-09NCT05371613
Infant Safe Sleep in Pediatric Emergency DepratmentDeath, Death, Sudden, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Infant DeathNot yet recruitingNot applicable2026-08-21NCT07779512
Long-term Follow-Up for RGX-121Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingNot stated2026-08-19NCT04597385
Supply Induced Demand in Intensive Care Unit: a Retrospective TrialDeath, Death, Sudden, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Infant DeathNot yet recruitingNot stated2026-08-17NCT07768410
Oral Azithromycin to Prevent Stillbirths and Infant Mortality in MaliDeath, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, pregnancy disorder, pregnancy disorder with abortive outcomeActive, not recruitingPhase 42026-08-07NCT03909737
Improving Pediatrician Counseling About Infant Safe Sleep Using the Electronic Medical RecordDeath, Death, Sudden, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Infant DeathRecruitingNot applicable2026-07-21NCT05503771
Walk With Me (WWM) for Perinatal GriefAbortion, Spontaneous, Death, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Infant Death, Miscarriage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Stress Disorders, Traumatic, Urogenital Diseases, acute stress disorder, post-traumatic stress disorder, pregnancy disorder, pregnancy disorder with abortive outcome, psychiatric disorderRecruitingNot applicable2026-07-15NCT07011940
Comparative Outcomes Related to Delivery-room Cord Milking In Low-resourced Kountries- PreTermCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Infant Death, Infant, Newborn, Diseases, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Premature Birth, Urogenital Diseases, anemia, hematologic disorder, neonatal anemia, pregnancy disorder, twin to twin transfusion syndromeRecruitingNot applicable2026-07-13NCT06240715
Perinatal Mortality Outcomes Through Household Infection PathwaysDeath, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Perinatal Death, Urogenital Diseases, pregnancy disorder, pregnancy disorder with abortive outcomeNot yet recruitingNot applicable2026-07-01NCT07620613
Identification of Genetic Variants Associated With Unexpected Infant Death SyndromeDeath, Death, Sudden, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Infant DeathRecruitingNot stated2026-06-18NCT06244433
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderEnrolling by invitationPhase 2 / Phase 32026-06-11NCT06075537
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 32026-06-04NCT07236606
Social Media and Risk-reduction Training for Preterm Infant Care PracticesDeath, Death, Sudden, Infant Death, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Infant DeathNot yet recruitingNot applicable2026-05-13NCT07420686
Advanced Wireless Sensors for Neonatal Care in the Delivery RoomAdverse Birth Outcomes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Female Urogenital Diseases and Pregnancy Complications, Infant Death, Infant, Newborn, Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, perinatal asphyxia, pregnancy disorderRecruitingNot applicable2026-05-08NCT06693817
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Gaucher disease, Infant Death, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis Type IV A, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, Wolman disease, brain disorder, central nervous system disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, sphingolipidosisRecruitingPhase 12026-03-17NCT04532047
Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal DemiseArrhythmias, Cardiac, Brugada syndrome, Cardiac Conduction System Disease, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Edema, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Fetal Diseases, Hemic and Lymphatic Diseases, Hernia, Hernia, Abdominal, Infant Death, Infant, Newborn, Diseases, Musculoskeletal Abnormalities, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Sudden Infant Death, Urogenital Diseases, alpha thalassemia spectrum, anemia, cardiovascular disorder, congenital heart disease, fetal erythroblastosis, gastroschisis, heart disorder, hematologic disorder, hereditary disease, hydrops fetalis, immune system disorder, inherited hemoglobinopathy, musculoskeletal system disorder, neonatal anemia, pregnancy disorder, pregnancy disorder with abortive outcome, thalassemia, twin to twin transfusion syndromeRecruitingNot stated2026-03-04NCT03775954
Can BCG Vaccination at First Health-facility Contact Reduce Early Infant Mortality?Death, Infant Death, Pathologic Processes, Pathological Conditions, Signs and SymptomsActive, not recruitingPhase 42026-02-11NCT04658680
Longitudinal Study of Neurodegenerative DisordersBone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, GM2 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis III, Neuronal Ceroid-Lipofuscinoses, Niemann-Pick disease, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pelizaeus-Merzbacher spectrum disorder, Sandhoff disease, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Tay-Sachs disease, X-linked disease, adrenoleukodystrophy, alpha-mannosidosis, bone disorder, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, demyelinating disease, disorder of methionine catabolism, giant axonal neuropathy 1, hereditary disease, histiocytosis, hypermethioninemia with deficiency of S-adenosylhomocysteine hydrolase, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, leukodystrophy, leukoencephalopathy with vanishing white matter 1, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 4, mucosulfatidosis, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, pantothenate kinase-associated neurodegeneration, purine nucleoside phosphorylase deficiency, sphingolipidosisRecruitingNot stated2026-02-09NCT03333200
Determining the Effect of Midwife-Led Safe Infant Care Training for Pregnant Women on Awareness of Sudden Infant Death Syndrome and Home AccidentsDeath, Infant Death, Pathologic Processes, Pathological Conditions, Signs and SymptomsNot yet recruitingNot applicable2026-01-20NCT07346482
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICBlood Protein Disorders, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dysgammaglobulinemia, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, leukocyte adhesion deficiency 1, leukocyte disorder, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, rheumatoid arthritis, severe combined immunodeficiency, sickle cell disease, sphingolipidosis, syndromic disease, thalassemiaRecruitingNot stated2026-01-13NCT04528355
Support Via Online Social Networks to Promote Safe Infant Care PracticesDeath, Infant Death, Pathologic Processes, Pathological Conditions, Signs and SymptomsRecruitingNot applicable2026-01-12NCT05767658
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTBone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dyskeratosis congenita, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, hereditary skin disorder, histiocytosis, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, juvenile idiopathic arthritis, leukocyte adhesion deficiency 1, leukocyte disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, severe combined immunodeficiency, sickle cell disease, skin disorder, sphingolipidosis, syndromic disease, thalassemiaRecruitingPhase 22025-12-15NCT01962415
Biological Impacts of Rising Temperatures on Maternal, Fetal, and Newborn Health: A Cohort Study (BIRTH Cohort)Adverse Birth Outcomes, Death, Female Urogenital Diseases and Pregnancy Complications, Heat Stress Disorders, Infant Death, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Premature Birth, Urogenital Diseases, injury, pregnancy disorderRecruitingNot stated2025-12-03NCT06957769